WASHINGTON, May 13, 2017 (GLOBE NEWSWIRE) — Intellia Therapeutics, Inc. (NASDAQ:NTLA), a leading genome editing company focused on the development of potentially curative therapeutics using CRISPR technology, presented an update on its long-term mouse genome editing and delivery studies and shared new, first-time data in rat models demonstrating consistent dose-dependent editing, at the American Society of Gene & Cell Therapy’s Annual Meeting (ASGCT).
These data, featured in a platform presentation on Saturday, May 13 at ASGCT showed:
- Six-month mouse study data demonstrating both durability and high editing efficiency in vivo, with approximately 70 percent editing at the target DNA site with a single intravenous dose. A 97 percent reduction of serum transthyretin (TTR) protein levels also was sustained.
- In addition, robust, dose-responsive lipid nanoparticle (LNP)-mediated editing of the TTR gene in rat livers following single intravenous administration; up to 66 percent editing at the target DNA site and up to 91 percent reduction in serum TTR protein levels.
- Both studies were conducted using Intellia’s proprietary LNP delivery system, providing high levels of liver delivery and rapid clearance.
“Data from the additional rat study further validates the in vivo CRISPR/Cas9 platform using Intellia’s proprietary LNP delivery system,” said David Morrissey, Ph.D., senior vice president, Platform and Delivery Technology. “In both species, we saw unprecedented in vivo liver editing results and consistent delivery of CRISPR/Cas9 with systemic administration using LNPs, while also showing the ability to expand our studies in larger species.”
About Intellia Therapeutics
Intellia Therapeutics is a leading genome editing company focused on the development of proprietary, potentially curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course. Our combination of deep scientific, technical and clinical development experience, along with our leading intellectual property portfolio, puts us in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Learn more about Intellia Therapeutics and CRISPR/Cas9 at intelliatx.com; Follow us on Twitter @intelliatweets.